An oral therapy for Huntington’s disease has been deemed eligible for a fast-track approval pathway by Australia’s medicines regulator.
The therapy, SKY-0515, is a small molecule designed to target RNA, the genetic instructions cells use to produce proteins involved in the disease.
Huntington’s disease is a rare inherited neurodegenerative disorder that causes progressive damage to brain cells and currently has no approved treatments that slow or stop its progression.
Australia’s medicines regulator, the Therapeutic Goods Administration (TGA), determined that SKY-0515 meets eligibility criteria for registration through its provisional approval pathway.
The drug is being developed by clinical-stage biotechnology company Skyhawk Therapeutics, which submitted its application for provisional approval to the TGA on the same day.
“We are grateful that the TGA has determined SKY-0515 for the treatment of Huntington’s disease meets its eligibility criteria for potential provisional approval,” said Bill Haney, Skyhawk’s chief executive.
“This represents an important first step toward what could be an accelerated path to approval in Australia and the world, offering the possibility of a disease modifying treatment for Huntington’s patients who urgently need therapies for this devastating disease.”
Treatment with SKY-0515 results in dose-dependent reductions of mHTT and PMS1, proteins linked to the development of Huntington’s disease.
In a phase 1C trial, patients receiving SKY-0515 showed a mean improvement of +0.64 points on the Composite Unified Huntington’s Disease Rating Scale (cUHDRS), a measure used to assess overall disease progression, at nine months.
This compares with an expected worsening of -0.73 points over the same period in symptomatic patients.
Huntington’s disease affects hundreds of thousands of people worldwide, including more than 40,000 symptomatic patients in the United States and over 2,000 in Australia.
The company has also launched a phase 2/3 trial called FALCON-HD, a randomised, double-blind, placebo-controlled, dose-ranging study evaluating SKY-0515 in 120 participants across 12 sites in Australia and New Zealand, and 400 participants at more than 40 sites worldwide.
Eligible patients will receive a once-daily oral dose at one of three dose levels or placebo for a treatment period of at least 12 months.
Enrolment in the phase 1C trial is now complete and the study is ongoing.
SKY-0515 is the first Skyhawk drug to enter clinical trials, and the company is developing additional therapies for neurological diseases where no approved disease-modifying treatments currently exist.
